Approximately 80% of rare diseases have a genetic etiology. Gene therapies hold the potential to transform the treatment of rare diseases and may provide a functional cure for some patients. However, these innovative new treatment options must come with equally innovative programs to optimize access in a global healthcare market that is still grappling with how to fairly assess their value and absorb their higher upfront costs. Nonetheless, the pipeline for gene therapies is burgeoning, with large pharmaceutical companies (e.g., Pfizer, Roche, Novartis) having an increasing stake in this arena. In this content, we will explore the clinical and commercial potential of gene therapies in key therapeutic areas (e.g., neurology, ophthalmology, hematology).
United States, EU5, Japan
More than 25 country-specific interviews with thought-leading neurologists, ophthalmologists, and hematologists. Supported by survey data collected for this and other Clarivate research.
Diagnosed prevalent and/or incident patient populations eligible for gene therapies in covered indications.
10-year, annualized, drug-level sales and patient share of key gene therapies through 2031, segmented by brands / generics, geography, and key indications.
Pipeline gene therapy coverage for select indications across therapy areas.
Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.
Disease Landscape & Forecast will feature continuous updates to provide timely insights and analyses as meaningful indication-specific news and events unfold.